WO2003054197A3 - A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism - Google Patents

A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism Download PDF

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Publication number
WO2003054197A3
WO2003054197A3 PCT/EP2002/014750 EP0214750W WO03054197A3 WO 2003054197 A3 WO2003054197 A3 WO 2003054197A3 EP 0214750 W EP0214750 W EP 0214750W WO 03054197 A3 WO03054197 A3 WO 03054197A3
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WO
WIPO (PCT)
Prior art keywords
library
modified
identification
structural genes
desired cell
Prior art date
Application number
PCT/EP2002/014750
Other languages
German (de)
French (fr)
Other versions
WO2003054197A2 (en
Inventor
Luca Perabo
Hildegard Buening
Joerg Enssle
Martin Ried
Michael Hallek
Nadja Huttner
Original Assignee
Medigene Ag
Luca Perabo
Hildegard Buening
Joerg Enssle
Martin Ried
Michael Hallek
Nadja Huttner
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Medigene Ag, Luca Perabo, Hildegard Buening, Joerg Enssle, Martin Ried, Michael Hallek, Nadja Huttner filed Critical Medigene Ag
Priority to CA2468882A priority Critical patent/CA2468882C/en
Priority to JP2003554898A priority patent/JP2005512569A/en
Priority to EP02787962.6A priority patent/EP1456383B1/en
Priority to AU2002352261A priority patent/AU2002352261B2/en
Priority to US10/498,163 priority patent/US20050106558A1/en
Priority to ES02787962.6T priority patent/ES2467156T3/en
Publication of WO2003054197A2 publication Critical patent/WO2003054197A2/en
Publication of WO2003054197A3 publication Critical patent/WO2003054197A3/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Communicable Diseases (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

This invention relates to a library of nucleic acids comprising a multiplicity of expressible structural genes, preferably cap genes, from an eukaryotic virus, preferably of a parvovirus.
PCT/EP2002/014750 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism WO2003054197A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
CA2468882A CA2468882C (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
JP2003554898A JP2005512569A (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid-modified particles useful for identifying virus clones with the desired cell orientation
EP02787962.6A EP1456383B1 (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
AU2002352261A AU2002352261B2 (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
US10/498,163 US20050106558A1 (en) 2001-12-21 2002-12-23 Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
ES02787962.6T ES2467156T3 (en) 2001-12-21 2002-12-23 A library of modified structural genes or modified capsid particles useful for the identification of viral clones with desired cell tropism

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US34413101P 2001-12-21 2001-12-21
US60/344,131 2001-12-21
US36234902P 2002-03-07 2002-03-07
US60/362,349 2002-03-07
US40711602P 2002-08-30 2002-08-30
US60/407,116 2002-08-30

Publications (2)

Publication Number Publication Date
WO2003054197A2 WO2003054197A2 (en) 2003-07-03
WO2003054197A3 true WO2003054197A3 (en) 2004-03-18

Family

ID=27407597

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2002/014750 WO2003054197A2 (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism

Country Status (7)

Country Link
US (1) US20050106558A1 (en)
EP (2) EP2363487A3 (en)
JP (1) JP2005512569A (en)
AU (2) AU2002352261B2 (en)
CA (1) CA2468882C (en)
ES (1) ES2467156T3 (en)
WO (1) WO2003054197A2 (en)

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DE19827457C1 (en) * 1998-06-19 2000-03-02 Medigene Ag Structural protein of AAV, its production and use
US20070172460A1 (en) * 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US20090191597A1 (en) * 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
EP2037892B1 (en) 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
US20100203083A1 (en) * 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2098536A1 (en) 2008-03-05 2009-09-09 4-Antibody AG Isolation and identification of antigen- or ligand-specific binding proteins
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
ES2531581T3 (en) 2010-10-21 2015-03-17 Deutsches Krebsforschungszentrum Reorientation of rat parvovirus H-1PV towards cancer cells by genetic manipulation of its capsid
KR20220056884A (en) 2011-04-22 2022-05-06 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Adeno-associated virus virions with variant capsid and methods of use thereof
EP2755677B1 (en) 2011-09-15 2020-11-04 Medigene AG Anti-her2 vaccine based upon aav derived multimeric structures
EP2692731A1 (en) * 2012-07-31 2014-02-05 Paul-Ehrlich-Institut Bundesamt für Sera und Impfstoffe Recombinant Adeno-Associated virus (AAV) vector particles displaying high-affinity ligands for cell-type specific gene delivery
CN104937100B (en) * 2012-12-25 2020-04-03 宝生物工程株式会社 AAV variants
ES2897508T3 (en) 2013-05-31 2022-03-01 Univ California Adeno-associated virus variants and methods of using them
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
SG10201810150UA (en) 2014-03-17 2018-12-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
EP3221456B1 (en) * 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
WO2016141078A1 (en) 2015-03-02 2016-09-09 Avalanche Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
WO2016154344A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
CN116286986A (en) 2016-07-29 2023-06-23 加利福尼亚大学董事会 Adeno-associated virus virions with variant capsids and methods of use thereof
WO2018075798A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CN110709511A (en) 2017-08-28 2020-01-17 加利福尼亚大学董事会 Adeno-associated virus capsid variants and methods of use thereof
RU2770922C2 (en) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Capsids of adeno-associated virus variants and methods of their application
US11851671B2 (en) * 2017-10-23 2023-12-26 Regents Of The University Of Minnesota Programmable assembly of virus composites for receptor-targeted gene delivery
CA3083472A1 (en) 2017-11-27 2019-05-31 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
CN112040988A (en) * 2018-04-27 2020-12-04 海德堡大学 Modified AAV capsid polypeptides for the treatment of muscle diseases
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CR20210444A (en) 2019-02-25 2021-11-02 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3715358A1 (en) 2019-03-28 2020-09-30 Universität zu Köln Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver

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Also Published As

Publication number Publication date
ES2467156T3 (en) 2014-06-12
AU2002352261B2 (en) 2008-02-07
US20050106558A1 (en) 2005-05-19
EP1456383B1 (en) 2014-03-12
CA2468882A1 (en) 2003-07-03
CA2468882C (en) 2015-09-29
AU2008202032B2 (en) 2012-09-20
AU2008202032A1 (en) 2009-11-26
EP2363487A3 (en) 2012-03-21
AU2002352261A1 (en) 2003-07-09
WO2003054197A2 (en) 2003-07-03
EP1456383A2 (en) 2004-09-15
EP2363487A2 (en) 2011-09-07
JP2005512569A (en) 2005-05-12

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